First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients

Baxalta now part of Shire0 sites17 target enrollmentSeptember 15, 2010

ConditionsHemophilia

Interventionsplacebo control ARC19499

Drugsplacebo control ARC19499

Overview

Phase: Phase 1
Intervention: placebo control
Conditions: Hemophilia
Sponsor: Baxalta now part of Shire
Enrollment: 17
Primary Endpoint: Pharmacokinetics (PK) of ARC19499
Status: Terminated
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Similar Trials

Overview

Brief Summary

The purpose of this study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia.

Registry

clinicaltrials.gov

Start Date

September 15, 2010

End Date

December 28, 2011

Last Updated

4 years ago

Study Type

Interventional

Study Design

Parallel

Sex

Male

Investigators

Sponsor

Baxalta now part of Shire

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Adult male patients ≥18 to ≤75 years of age.
•Hemophilia of any type or severity.
•Patients who are negative for hepatitis B surface antigen (HBsAg), and human immunodeficiency virus (HIV) I and II antibody tests at screening.
•Male patients who, with their partners, are willing to use 2 effective, methods of contraception (i.e., for both self and partner) throughout the study and for at least 3 months after discontinuation of study drug treatment.
•All patients must be capable of understanding and complying with the protocol and must have signed the informed consent document.

Exclusion Criteria

•Female patients;
•If on a prophylactic coagulation factor concentrate regimen, inability or unwillingness to discontinue prophylaxis during participation in this study.
•Existence of other co-existing bleeding disorder (e.g., von Willebrand Disease).
•Medical history of venous or arterial thromboembolism.
•Scheduled for elective surgical procedure during the conduct of this study.
•Use of an investigational drug within 30 days of study entry.
•Transaminase values \> 3 x upper limit of normal (ULN) at time of screening.
•Haemoglobin \<12.0 g/dL.
•Participants who, in the opinion of the Investigator, have a significant infection or known inflammatory process on screening.
•Participants who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders.

Arms & Interventions

saline for injection

Intervention: placebo control

ARC19499 Low Dose

Intervention: ARC19499

ARC19499 Mid Dose

Intervention: ARC19499

ARC19499 High Dose

Intervention: ARC19499

Outcomes

Primary Outcomes

Pharmacokinetics (PK) of ARC19499

Time Frame: 2 weeks

The PK profile of ARC19499 administered by single and multiple subcutaneous injections will be characterized. The bioavailability of subcutaneously injected ARC19499 relative to that of intravenously infused ARC19499 will be determined.